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Research programme: gene therapies - Voyager Therapeutics

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Drug Profile

Research programme: gene therapies - Voyager Therapeutics

Alternative Names: AAV Frataxin; AAV-delivered gene therapies for CNS disorders - Voyager Therapeutics; AAVrh.10-vectored PHF1; ALS SOD1; Amyotrophic lateral sclerosis gene therapy - Voyager Therapeutics; CNS-Directed GBA1 Gene Therapy - Voyager Therapeutics; Friedreich’s ataxia gene therapy - Voyager Therapeutics; GBA1 gene replacement therapy - Voyager Therapeutics; Tau silencing gene therapy - Voyager Therapeutics; VY FXN01; VY SMN101; VY SOD102; VY-NAV01; VY-SOD101

Latest Information Update: 05 Mar 2024

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At a glance

  • Originator Voyager Therapeutics
  • Class Analgesics; Antidementias; Gene therapies; Neuroprotectants; Small interfering RNA
  • Mechanism of Action Gene transference; Nav1.7 voltage-gated sodium channel inhibitors; Tau protein expression inhibitors; Tau protein inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Huntington's disease
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Alzheimer's disease; CNS disorders; Friedreich's ataxia
  • No development reported Dementia; Huntington's disease; Pain; Spinal muscular atrophy
  • Discontinued Amyotrophic lateral sclerosis

Most Recent Events

  • 05 Mar 2024 Voyager Therapeutics plans a phase I trial for Amyotrophic lateral sclerosis (Parenteral)
  • 28 Feb 2024 Voyager anticipates an IND filing for Amyotrophic lateral sclerosis in mid of 2025
  • 23 Feb 2024 Preclinical development is ongoing USA

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